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Article discusses this new drug’s progress through the clinical trials, and its promise as of its phase 2 trials on patients with cystic fibrosis. It further details the American Journal of Respiratory Cell and Molecular Biologyn research which shows that daily long-term treatment of the cells of cystic fibrosis with ow doses of miglustat appears to correct the pathological abnormalities and have promise insofar as effective treatment is concerned.

Consists of basic information about Orfadin, generically known as nitisinone when it was approved and for what uses. as wel ca clinical results and evaluations. Also discusses the mechanism of action, which is that it is a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase.